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Jain et al. Rare Dis Orphan Drugs J 2024;3:8  https://dx.doi.org/10.20517/rdodj.2023.42  Page 5 of 9

                Malaysia  33.94  No formal legislation          Allocation of approximately 5.3 million US$ has   [37,38]
                                 Health Ministry’s National Strategic Plan for   supported 60 patients thus far
                                 Medical Programmes currently under review  Private insurance coverage available for some RD
                                                                Orphan medicine designation under the purview of
                                                                NPRA
                Nepal  30.55     No specific legislation on RD  Congenital disorders, because of their rarity, have   [39,40]
                                                                not been addressed at the health policy level
                Australia  25.98  2020 National Strategic Action Plan for RDs (the  Most medicines subsidized through PBS    [41-45]
                                 Action Plan) launched          RDs that are rejected by PBS on cost-effectiveness
                                 The Pharmaceutical Benefits Scheme (PBS)   grounds are supported by the 1995 Life Saving
                                 enables free medicines for 139 ‘life-saving and   Drugs Program
                                 disease prevention’ medicines
                Taiwan  23.32    The RD and Orphan Drug Act was approved in   The Rare Disease and Orphan Drug Act provides   [46-48]
                                 2000. The Act defines RD, and facilitates   grants, fast-track approval, protocol assistance, and
                                 research, access of orphan drugs and special   market exclusivity
                                 nutritional foods. The Act enables the prevention
                                 and early diagnosis of RDs
                Hong Kong 7.35   No RD legislation              Clinical diagnosis and assessments, multi-  [6,49,50]
                                                                disciplinary care and rehabilitation services, and
                                                                subsidized drugs are available by the Hospital
                                                                Authority, a statutory board that manages all public
                                                                hospitals.
                                                                Designated Orphan Drugs are covered by the
                                                                government's annual financial budget allocated to
                                                                the Hospital Authority and complimented by the
                                                                Samaritan Fund (51 drugs) and the Community
                                                                Care Fund Medical Assistance Programme (37
                                                                drugs) since 2021 for means-tested patients
                Singapore  5.64  No formal RD legislation or definition of RD.   The Rare Disease Fund, a national multi-  [51-54]
                                 Criteria for accessing the Rare Disease Fund   stakeholder charity fund set up in 2019, has
                                 stipulates rare as < 4/10,000 (i.e., < 1,600   provided financial support to 9 patients (as of
                                 people in Singapore). However, since the fund   March 2023) for 7 medicines approved for 5 RDs
                                 only covers ultra-rare diseases currently, the
                                 definition for ultra-rare is < 2 in 50,000 people
                                 (i.e., < 225 people with the condition in
                                 Singapore)
                                 Health Products Act governs orphan drugs as
                                 part of all therapeutic products
                New    5.12      No specific policy on rare disorders  The Pharmaceutical Management Agency   [55-61]
                Zealand                                         (Pharmac) decides on behalf of Health New
                                                                Zealand (Te Whatu Ora) which medicines and
                                                                pharmaceutical products will be subsidized. The
                                                                Pharmac Rare Disorders Advisory Committee
                                                                evaluates funding applications and makes
                                                                recommendations to the Pharmacology and
                                                                Therapeutics Advisory Committee (PTAC) and
                                                                Pharmac

               RD: Rare diseases; CoE: centre of excellence; PLWRD: persons living with a rare disease.


               Malaysia in 2023. APARDO has also organized a series of webinars on diverse topics, such as Essential
               Medicines and Cell and Gene Therapy. It has also partnered with organizations such as rare diseases
               international (RDI) for their initiative, the Global Network for RDs, and regionally, through support
               towards the RD Network, previously associated with the Asian Pacific Economic Cooperation (APEC)
               program. Over the years, the organization has collected data on patient needs and gaps and contributed to
               publications [8,48,62] . As a member of various global organizations such as RDI and the International Rare
               Diseases Research Consortium (IRDiRC), APARDO continues to contribute perspectives from the APAC
               region with the goal of improving the lives of PLWRD and their families.


               Opportunities for strengthened collaborations
               The hyper diversity of languages, cultures, paternalistic healthcare systems, and sparsity of developed
               patient organizations pose an enormous challenge for effective and efficient exchanges across APAC.
               Territorial differences ranging from vast areas (China, India, Australia) to thousands of islands (Indonesia,
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